Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Children's Hospital of Philadelphia
Age
8–16
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Age ≥ 8 years; \<16 years
Written informed consent provided
Balletic Guanine-adenine-adenine (GAA) trinucleotide repeat length \> 55 in intron 1 of Frataxin (FXN) and/or GAA repeat length \> 55 in intron 1 of FXN in one allele and another type of mutation that is inferred to cause loss of function in the second FXN allele as documented in the medical record
Friedreich's Ataxia Rating Scale (FARS) Functional staging score of ≤ 5\^ and total modified Friedreich's Ataxia Rating Scale (mFARS) score of ≤ 65 on enrolment
You may not be if
Age \< 8 years \> 16 years
Acute or ongoing medical or other conditions that is deemed to interfere with the conduct and assessments of the study
Other psychiatric or neurologic conditions apart from FRDA that, in the opinion of the Site Investigator, would interfere with the conduct and assessments of the study
MR contraindications (e.g., pacemaker or other metallic surgical implants)
Presence of metallic dental braces
Currently pregnant participants
Confined to wheelchair or bed with total dependency for all activities of daily living. Total disability.
